Nov 11, 2025: Cereno Scientific (Nasdaq First North: CRNO B), an innovative biotech pioneering treatments to enhance and extend life for people with rare cardiovascular and pulmonary diseases, today announced the submission of the clinical trial protocol for the planned global Phase IIb trial of its lead drug candidate CS1 to the U.S. Food and Drug Administration (FDA). The submission marks an important milestone, moving the company closer to advancing CS1 into its next clinical phase and toward bringing a novel therapeutic approach to patients living with pulmonary arterial hypertension (PAH).
The planned Phase IIb trial is designed to further evaluate the safety, tolerability and efficacy of CS1, a histone deacetylase inhibitor (HDACi) in development as an oral treatment targeting the root mechanisms of PAH through epigenetic modulation. The Phase IIb trial will build on the results from the completed Phase IIa trial, where CS1 demonstrated a favorable safety and tolerability profile together with encouraging efficacy signals including reverse vascular remodeling, improved right heart function, and enhanced patient quality of life. The new global, multicenter, placebo-controlled trial will be conducted in collaboration with a leading international contract research organization (CRO). Regulatory interactions in other key regions will follow as part of the global start-up preparations.
“The submission of the protocol marks a major milestone and underscores the focus, commitment and outstanding collaboration across our team and global CRO,” said Rahul Agrawal, CMO and Head of R&D of Cereno Scientific. “This protocol has been developed in alignment with the FDA’s feedback from our recent Type C meeting, and we now look forward to the agency’s review ahead of initiating the trial.”
PAH is a rare, progressive and life-threatening disease characterized by high blood pressure in the pulmonary arteries that leads to right heart failure and premature death. Current standard treatments mainly focus on managing symptoms, leaving a significant unmet need for disease-modifying therapies that can change the course of disease and improve long-term outcomes.
“This milestone moves us closer to our goal of delivering a first-in-class therapy that targets the root mechanisms of PAH through epigenetic modulation,” said Sten R. Sörensen, CEO of Cereno Scientific. “With CS1, we have an opportunity to potentially change the treatment paradigm for patients facing this devastating disease and, in doing so, create meaningful long-term benefits for both patients and shareholders.”
Following the FDA’s standard 30-day review, Cereno Scientific anticipates clearance to proceed with the trial. The Phase IIb trial is planned to begin during H1 2026 as part of the company’s global development program for CS1.